amyotrophic lateral sclerosis

FDA Approves New Medication for Treatment of ALS

The FDA has approved sodium phenylbutyrate/taurursodiol (Relyvrio) as a treatment option for patients diagnosed with amyotrophic lateral sclerosis (ALS).

The approval of sodium phenylbutyrate/taurursodiol follows a 24-week, multicenter, randomized, double-blind, placebo-controlled, parallel-group study that demonstrated the efficacy of the treatment. During the trial, 137 adult patients with ALS were randomized to either receive the treatment or a placebo. The patients who received sodium phenylbutyrate/taurursodiol showed a slower rate of decline on a clinical assessment of daily functioning when compared to individuals receiving a placebo.

Further, patients who received treatment had a longer overall survival compared to patients who received a placebo.

Sodium phenylbutyrate/taurursodiol is medication that can be taken orally by combining 1 packet in 8 ounces of room temperature water. Additionally, the medication can be administered through a feeding tube. The medication has a recommended dosage of 1 packet (3 grams sodium phenylbutyrate and 1-gram taurursodiol) for the first 3 weeks daily. Physicians will increase the dosage by 1 packet twice a day after the initial 3 weeks.

Common adverse effects include diarrhea, abdominal pain, nausea, and upper respiratory tract infection. Taurursodiol is a bile acid that may cause worsening diarrhea in patients with disorders that interfere with the circulation of bile acid.

“This approval provides another important treatment option for ALS, a life-threatening disease that currently has no cure,” Billy Dunn, MD, director of the Office of Neuroscience in the FDA’s Center for Drug Evaluation and Research said in a press release. “The FDA remains committed to facilitating the development of additional ALS treatments.”


—Jessica Ganga


FDA approves new treatment option for patients with ALS. News release. US Food and Drug Administration; September 29, 2022. Accessed October 7, 2022.